ES2962439T3 - Agente terapéutico que activa la función mTORC1 para su uso en el tratamiento de la enfermedad de Huntington - Google Patents

Agente terapéutico que activa la función mTORC1 para su uso en el tratamiento de la enfermedad de Huntington Download PDF

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Publication number
ES2962439T3
ES2962439T3 ES15876243T ES15876243T ES2962439T3 ES 2962439 T3 ES2962439 T3 ES 2962439T3 ES 15876243 T ES15876243 T ES 15876243T ES 15876243 T ES15876243 T ES 15876243T ES 2962439 T3 ES2962439 T3 ES 2962439T3
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ES
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Prior art keywords
aav
therapeutic agent
mice
rhes
cell
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ES15876243T
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English (en)
Spanish (es)
Inventor
Beverly Davidson
John Lee
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University of Iowa Research Foundation UIRF
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University of Iowa Research Foundation UIRF
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    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46Hydrolases (3)
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/0012Galenical forms characterised by the site of application
    • A61K9/0085Brain, e.g. brain implants; Spinal cord
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/28Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N7/00Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y306/00Hydrolases acting on acid anhydrides (3.6)
    • C12Y306/05Hydrolases acting on acid anhydrides (3.6) acting on GTP; involved in cellular and subcellular movement (3.6.5)
    • C12Y306/05002Small monomeric GTPase (3.6.5.2)
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Medicinal Chemistry (AREA)
  • Organic Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Genetics & Genomics (AREA)
  • Epidemiology (AREA)
  • Biomedical Technology (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • Immunology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Molecular Biology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Psychiatry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Hospice & Palliative Care (AREA)
  • Virology (AREA)
  • Psychology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Microbiology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
ES15876243T 2014-12-30 2015-12-30 Agente terapéutico que activa la función mTORC1 para su uso en el tratamiento de la enfermedad de Huntington Active ES2962439T3 (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201462098085P 2014-12-30 2014-12-30
PCT/US2015/068034 WO2016109649A1 (en) 2014-12-30 2015-12-30 Methods and compositions for treating brain diseases

Publications (1)

Publication Number Publication Date
ES2962439T3 true ES2962439T3 (es) 2024-03-19

Family

ID=56285031

Family Applications (1)

Application Number Title Priority Date Filing Date
ES15876243T Active ES2962439T3 (es) 2014-12-30 2015-12-30 Agente terapéutico que activa la función mTORC1 para su uso en el tratamiento de la enfermedad de Huntington

Country Status (12)

Country Link
US (1) US11612641B2 (pt)
EP (1) EP3240577B1 (pt)
JP (1) JP6782701B2 (pt)
KR (1) KR102618947B1 (pt)
CN (1) CN107405414A (pt)
AU (1) AU2015374043B2 (pt)
BR (1) BR112017013674A2 (pt)
CA (1) CA2971687A1 (pt)
ES (1) ES2962439T3 (pt)
HK (1) HK1245127A1 (pt)
SG (1) SG11201704829QA (pt)
WO (1) WO2016109649A1 (pt)

Families Citing this family (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US10597660B2 (en) 2014-11-14 2020-03-24 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (ALS)
ES2962439T3 (es) 2014-12-30 2024-03-19 Univ Iowa Res Found Agente terapéutico que activa la función mTORC1 para su uso en el tratamiento de la enfermedad de Huntington
EP3458589A4 (en) 2016-05-18 2020-01-01 Voyager Therapeutics, Inc. COMPOSITIONS AND METHODS FOR TREATING HUNTINGTON'S DISEASE
KR20240056729A (ko) 2016-05-18 2024-04-30 보이저 테라퓨틱스, 인크. 조절성 폴리뉴클레오티드
CL2016003282A1 (es) * 2016-12-21 2017-08-18 Univ Chile Virus aav/igf2, método de tratamiento genético y su uso en enfermedades relacionadas con mal plegamiento de proteínas tal como la enfermedad de huntington
WO2018132791A1 (en) 2017-01-16 2018-07-19 The Regents Of The University Of California Treatment of neurodegenerative conditions by disruption of rhes
JP2020518259A (ja) 2017-05-05 2020-06-25 ボイジャー セラピューティクス インコーポレイテッドVoyager Therapeutics,Inc. ハンチントン病治療組成物および方法

Family Cites Families (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20050255086A1 (en) * 2002-08-05 2005-11-17 Davidson Beverly L Nucleic acid silencing of Huntington's Disease gene
US20050009112A1 (en) 2003-03-07 2005-01-13 Fred Hutchinson Cancer Research Center, Office Of Technology Transfer Methods for identifying Rheb effectors as lead compounds for drug development for diabetes and diseases associated with abnormal cell growth
WO2006116716A2 (en) 2005-04-27 2006-11-02 University Of Florida Materials and methods for enhanced degradation of mutant proteins associated with human disease
KR101286870B1 (ko) * 2009-11-30 2013-07-16 (주)아모레퍼시픽 황기 추출물을 포함하는 대사 촉진 조성물
MX340452B (es) * 2011-10-28 2016-07-08 Novartis Ag Novedosos derivados de purina y su uso en el tratamiento de enfermedades.
AU2012332471A1 (en) * 2011-11-01 2014-05-01 Children's Medical Center Corporation Co-activation of mTOR and STAT3 pathways to promote neuronal survival and regeneration
WO2013070976A1 (en) 2011-11-08 2013-05-16 Intellikine, Llc Treatment regimens using multiple pharmaceutical agents
WO2013116691A1 (en) * 2012-02-02 2013-08-08 The Washington University Methods for improving muscle strength
KR101626526B1 (ko) 2014-10-30 2016-06-02 경북대학교 산학협력단 hRheb를 유효성분으로 포함하는 허혈성 뇌질환의 예방 또는 치료용 조성물
ES2962439T3 (es) 2014-12-30 2024-03-19 Univ Iowa Res Found Agente terapéutico que activa la función mTORC1 para su uso en el tratamiento de la enfermedad de Huntington

Also Published As

Publication number Publication date
JP2018502112A (ja) 2018-01-25
JP6782701B2 (ja) 2020-11-11
EP3240577A1 (en) 2017-11-08
US11612641B2 (en) 2023-03-28
CA2971687A1 (en) 2016-07-07
KR102618947B1 (ko) 2023-12-27
KR20170098931A (ko) 2017-08-30
EP3240577B1 (en) 2023-07-05
CN107405414A (zh) 2017-11-28
AU2015374043A1 (en) 2017-07-13
BR112017013674A2 (pt) 2018-02-06
US20180000907A1 (en) 2018-01-04
EP3240577A4 (en) 2018-07-25
WO2016109649A1 (en) 2016-07-07
HK1245127A1 (zh) 2018-08-24
SG11201704829QA (en) 2017-07-28
AU2015374043B2 (en) 2021-05-13

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